Resources

Biotech Companies Urge Congress to Pass Bipartisan ORPHAN Cures Act

H.R. 5539 ORPHAN Cures Act

S. 3131 ORPHAN Cures Act

Task Force ORPHAN Cures Act Support Letter

Task Force ORPHAN Cures Act One-Pager

Task Force press release

Biotechnology Industry Organization: ORPHAN Cures Act One-Pager

Biotechnology Industry Organization: ORPHAN Cures Act a “welcome and bipartisan fix” to the IRA’s negative impact on drug development

California Life Sciences: California Life Sciences Urges Congress to Support Rare Disease Patients

Rare Disease Company Coalition: New Bill Protects Continued Development for Rare Disease Medicines

Inflation Reduction Act Orphan Drug Exclusion –42 U.S. Code § 1320f–1(e)(3)(a)

CRS Report: Implementation of the Medicare Drug Price Negotiation Program: Centers for Medicare and Medicaid Guidance and Legal Considerations

CMS: Memorandum, Implementation of Sections 1191 – 1198 of the Social Security Act for Initial Price Applicability Year 2026, and Solicitation of Comments

CMS: Revised Guidance, Implementation of Sections 1191 – 1198 of the Social Security Act for Initial Price Applicability Year 2026

CMS: Negotiation Guidance Published Comments

FDA: Rare Diseases – Frequently Asked Questions

FDA Orphan Designations – Worldwide Orphan Medicinal Designations Workshop

FDA: Orphan Drug Act – Relevant Excerpts

The New England Council: Support of S. 3131 and H.R. 5539

Biotechnology Industry Organization: Comments on Medicare Drug Price Negotiation Program Initial Guidance

Biotechnology Industry Organization: New CMS guidance a ‘missed opportunity’ for future medical innovation, patients

EveryLife: Comments on Medicare Drug Price Negotiation Program Initial Guidance

National Health Council: Comments on Medicare Drug Price Negotiation Program Initial Guidance

National Organization for Rare Disorders: Comments on Medicare Drug Price Negotiation Program Initial Guidance

National Organization for Rare Diseases: Final Inflation Reduction Act Guidance Risks Stifling Innovation for Rare Disease Community

National Organization for Rare Disorders: Letter to CMS from Rare Disease Patient Organizations – Medicare Drug Price Negotiation Program Guidance

AstraZeneca Complaint

Note: This material is included for informational purposes only. The Task Force is not a party to or involved with this or any other Medicare Drug Price Negotiation litigation.

Government Response to the Complaint

Real Clear Health: A Small Fix to the IRA Will Give Hope to Patients With Rare Diseases

The Hill: The Inflation Reduction Act adds new barriers to curing rare disease

BioCentury: IRA’s unintended consequences include harm to rare disease patients

Biotechnology Industry Organization: The Outlook for Orphan Drugs

Council for Affordable Health Coverage: How the Inflation Reduction Act is Impacting Rare Disease Patients

First Opinion in STAT News: The IRA needs changes to better support patients with rare diseases

Health Affairs: How The IRA Could Delay Pharmaceutical Launches, Reduce Indications, And Chill Evidence Generation

Health Capital Group: Rare Disease Companies in the Public Markets: Challenging Performance Against a Backdrop of Policy Uncertainty

Incubate: Life Sciences Investment Tracker

Pharmaceutical Research and Manufacturers of America: Reduction Act stifles innovation for small molecule medicines

Pharmaceutical Technology: Rare disease drug development needs regulatory incentives to grow in 2024

Rare Access Action Project: Inflation Reduction Act to Harm Rare Disease Innovation

Tax Foundation: Inflation Reduction Act’s Price Controls Are Deterring New Drug Development

Tufts University: Follow-On Indications for Orphan Drugs Related to the Inflation Reduction Act

University of Chicago: The Potentially Larger Than Predicted Impact of the IRA on Small Molecule R&D and Patient Health

VitalTransformation: IRA’s Impact on the US Biopharma Ecosystem